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Prophylactic oral bisphosphonate therapy in duchenne muscular dystrophy

Lookup NU author(s): David Rawlings, Dr Claire Wood, Professor Timothy Cheetham, Dr Cecilia Jimenez MorenoORCiD, Dr Anna Mayhew, Dr Michelle Eagle, Professor Michela GuglieriORCiD, Professor Volker StraubORCiD, Emerita Professor Katherine Bushby, Dr Anna Sarkozy


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IntroductionWe assessed prophylactic use of bisphosphonate (BP) in Duchenne muscular dystrophy (DMD) patients on glucocorticoid (GC) therapy.MethodsFifty-two DMD patients on daily GC were offered BP (oral risedronate). Patients were reviewed for tolerability, side effects, bone pain, and fracture frequency. Bone mineral density (BMD) was determined by annual dual energy X-ray absorptiometry. BP-treated patients were compared with 15 BP-naive patients (untreated cohort).ResultsSide effects occurred in 9 patients. Thirty-six patients continued BP therapy for over 12 months (mean, 3.6 years). Five treated patients reported bone pain. Three treated patients suffered a vertebral fracture, significantly less than in the untreated cohort (5/15). Lumbar spine adjusted BMD Z-scores remained unchanged in treated patients and were significantly greater than in the untreated cohort.ConclusionsProphylactic oral risedronate therapy was tolerated by most DMD patients. It appears to maintain BMD and may reduce fracture rate in DMD patients on GC. Muscle Nerve54: 79-85, 2016

Publication metadata

Author(s): Srinivasan R, Rawlings D, Wood CL, Cheetham T, Moreno ACJ, Mayhew A, Eagle M, Guglieri M, Straub V, Owen C, Bushby K, Sarkozy A

Publication type: Article

Publication status: Published

Journal: Muscle & Nerve

Year: 2016

Volume: 54

Issue: 1

Pages: 79-85

Print publication date: 01/07/2016

Online publication date: 27/04/2016

Acceptance date: 20/11/2015

ISSN (print): 0148-639X

ISSN (electronic): 1097-4598

Publisher: Wiley-Blackwell


DOI: 10.1002/mus.24991


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Funder referenceFunder name
MR/K000608/1Medical Research Council